Advocates applaud Sask. for funding high-cost therapy

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When Lindsay Williamson’s baby Mason was diagnosed with a rare neuromuscular disorder, she pondered three options: fundraise, win a lottery or move.

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Those are the heartbreaking choices some families with spinal muscular atrophy have to make in order to access advanced gene therapies. One costs nearly $3 million, making it among the most expensive treatments in the world.

On Thursday, Saskatchewan’s government agreed to pay for that therapy, sold under the name Zolgensma, and another medication, Evrysdi, for eligible children in the province.

Williamson hopes the decision will spare other parents.

“What matters in the end is we have received this care now, and other families will as well,” she said.

Spinal muscular atrophy, or SMA, is a rare genetic condition that causes degradation of muscle in the limbs. Over time, it can also affect muscles needed to breathe and swallow. Some versions start in infancy, others later in life. Some are fatal, whereas other patients live well into their adult years.

Saskatchewan’s coverage of Zolgensma and Evrysdi comes roughly two years after it agreed to cover Spinraza, another high-cost gene therapy for SMA.

Desiree Parisien, a Saskatchewan resident with Cure SMA Canada, began taking Spinraza about two and a half years ago. She hoped the medication, administered four times a year via a spinal tube, would slow the progression of the disease so she could spend time with her husband and child, she said.

“It was more so, if I can hug them until I’m 60 and 70, that’d be great.”

The results exceeded expectations. Parisien said she regained muscle function she had been losing since she was a young girl. She was able to roll over, move to her knees and sit up without help.

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“There are no words to express what it feels like to have gone from not being able to do something for decades to being able to do it again,” Parisien said.

Dr. Kerri Schellenberg, a neuromuscular neurologist and the medical director of the ALS and Neuron Muscle Clinic in Saskatoon, said adult patients have regained function in a similar way since starting such therapies, which she described as life-changing.

“These medications have completely altered the outcome of SMA. I can’t overstate that enough,” Schellenberg said.

They come at a steep price. Saskatchewan estimates roughly one in 10,000 newborns has SMA. The small size of the patient group, the advanced nature of the therapy and the massive cost of clinical trials means costs are sky-high.

Saskatchewan’s Ministry of Health estimates a single dose of Zolgensma, a one-time therapy, costs $2.9 million, though the province may pay less under a confidential pharmaceutical agreement it brokers with other provinces. It estimates about one newborn a year may be eligible for the medication. The province recently announced it will screen all newborns for SMA, which advocates say will help catch it early.

Evrysdi, approved for infants, children and young adults, is listed at between $90,000 and $350,000 per patient depending on weight, outside the confidential pharmaceutical deal. The ministry anticipates as many as 10 people may receive that therapy each year.

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Advocates like Parisien have fought across the country to expand access to the medications, but Williamson said coverage varies between provinces. That leaves some parents to fundraise the cost themselves, or even move. Novartis, the company behind Zolgensma, even held a lottery for parents who otherwise can’t afford it.

“Postal code health care is real in Canada. People don’t think it exists,” Williamson said. The treatment gave her family the “gift of time” with their newborn baby, she added.

Parisien said she’s happy knowing she won’t have to help another family with a young child find a way to pay the cost of life.

“We are so fortunate to be in Saskatchewan, even though it’s cold as hell.”

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